Status:
ENROLLING_BY_INVITATION
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
Lead Sponsor:
Thomas Damgaard Sandahl
Collaborating Sponsors:
Ultragenyx Pharmaceutical Inc
Conditions:
Wilson Disease
Eligibility:
All Genders
18+ years
Brief Summary
The primary objective of this study is to investigate the effect of gene therapy (UX704) on copper distribution and excretion in Wilson disease patients. The effect is investigated using 64Cu positron...
Detailed Description
Patients entering this study are included from the ongoing clinical study "A Phase 1/ 2/ 3 Study of UX701 Gene Therapy in Adults With Wilson Disease" (NCT04884815). The patients will have received gen...
Eligibility Criteria
Inclusion
- Patients with Wilson disease who have received gene therapy (UX701) as part of NCT04884815
- Age above 18 years
- Women of childbearing potential must accept to use safe contraception at the time of the PET scans: Spiral or hormonal contraception (oral pills, implants, transdermal patches, vaginal rings, or depot injections)
- At the day of the first PET scan, a negative urine pregnancy test is required for women of childbearing potential
- Before the first PET scan, a signed informed consent must be completed
Exclusion
- Known hypersensitivity to Cu-64, copper in general, or other components of the radiotracer formulation
- Claustrophobia
- Pregnancy, breastfeeding, or plans to become pregnant
Key Trial Info
Start Date :
July 8 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 1 2026
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT07159581
Start Date
July 8 2025
End Date
January 1 2026
Last Update
September 8 2025
Active Locations (1)
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1
Aarhus University Hospital
Aarhus, Aarhus N, Denmark, 8200